The waiting game

For the past 45 days, we have been waiting to hear if the FDA was going to approve NNZ-2566 with Orphan Drug Status. Everyday I (Jenny) have thought about it anxiously, even on days I tried not to think about it, I thought about it. I stalked the Rett family pages, I stalked parents that put their girls through the trial, I stalked Neuren (the pharmaceutical company). Sixty days doesn’t seem like a long time, unless you are waiting to hear about a drug that could possibly change the course of your daughter’s life for the better. During the past 45 days, 4 girls with Rett Syndrome passed away. So yes, it is extremely hard not to think about a drug that could change these girls lives and the lives of so many families. I felt so much anger towards Rett, with each girl that passed away, I kept thinking we are so close, but not close enough for so many. Most recently, the little girl who passed away was relatively healthy, went to bed and just didn’t wake up. It is a reality that we face and it is truly a nightmare. When people say to me, “Oh that is my worst nightmare”. No kidding! We are living it. This is why we want drugs to be approved by the FDA sooner than later, this is why we need clinical trials, this is why we fundraise to try to get there.

So yesterday I woke up and saw the first post that stated the NNZ-2566 drug, which we will now call Trofinetide, was give Orphan drug status approval. I cried. Happy tears, sad tears and out of relief. One step closer. With each step we are closer to a treatment and it really is amazing. We have only been on this Rett journey for 15 months. We have only really seen the effects of Rett Syndrome this past year. (Maggie had delays for about 6 months prior to diagnosis but not really any symptoms of Rett) And I can tell you, it is exhausting, stressful, and occasionally nightmarish. To have hope this early in our journey is fortunate. I have other parents tell me quite often how strong they think AJ and I are and that they admire us, which is very nice. Imagine how we feel towards those parents with older Rett girls, who have been fighting longer, harder and at the same time fundraising and putting their girls in trials in order to get here. They deserve this drug,Trofinetide.

We don’t really know what the next step is for Trofinetide. I’m pretty sure it is a Pediatric trial phase. But we do know, we are one step closer. For Rett families, progress feels amazing, because we have seen enough regression.

Here is a video from Neuren explaining how Trofinetide works…click here. Also at 7minutes 38 seconds they explain how it works on a molecular level for Rett. Do we know what symptoms improved? Not really. The families that are in the trial can’t tell us yet, but when we know, you’ll know.

But seriously guys, this is a huge step in the right direction. Maggie could have a treatment in her childhood. My best guess is 2-3 years. I’m not a scientist or having any information, its just my guess. Let’s hope I’m the world’s best guesser.